AMT-130 (ifezuntirgene inilparvovec) for Huntington's disease
The FDA is expected to decide on AMT-130 for Huntington's disease in an undisclosed window, under a BLA from uniQure.
What is being decided
uniQure has a BLA under FDA review for AMT-130 (ifezuntirgene inilparvovec) in Huntington's disease. One-time gene therapy delivered by neurosurgery into the striatum to silence the huntingtin gene; would be the first treatment shown to slow Huntington's disease. uniQure submitted the BLA for accelerated approval on September 2, 2026 and requested priority review; the FDA's filing decision is due within 60 days and no target action date has been disclosed. Holds Breakthrough Therapy, RMAT and Fast Track designations.
Who this decision matters to
Huntington disease is an autosomal dominant neurodegenerative disorder caused by an expanded CAG trinucleotide repeat in the huntingtin gene. The progressive disease causes movement problems, cognitive decline, and psychiatric symptoms. Adult-onset disease typically appears ages 30-50, with juvenile-onset forms presenting earlier with different symptom profiles. Multiple disease-modifying therapies are in clinical trials.
Prevalence: 5-10 per 100,000 people of European descent; lower in other populations. See our full Huntington Disease page for current treatments, recruiting trials, and community resources.
What each outcome would mean
An approval starts a second race rather than ending the first one: specialty pharmacy setup, insurance review, and patient assistance typically take weeks even when everything goes right. Our guide to the 90 days after a rare disease approval explains the timeline and the moves families can make on day 1.
A complete response letter would mean the FDA declined to approve in the application's current form. CRLs are often about manufacturing or data presentation rather than efficacy, and resubmission is common. Either way, this page updates with the outcome and what it means.
Where Huntington Disease treatment stands today
Huntington disease research is advancing 3 distinct approaches to lower mutant huntingtin levels. Antisense oligonucleotides (ASOs) like tominersen reduce HTT mRNA through intrathecal injection. Gene therapies like AMT-130 use viral vectors to deliver a corrected gene directly to the brain. Splicing modifiers like votoplam (PTC518) are oral drugs that alter HTT pre-mRNA splicing. Each approach has different trial designs and eligibility requirements. Genetic testing to confirm your CAG repeat length is the first step for any HD trial. Premanifest gene carriers (CAG 40+ with no motor symptoms) have a growing number of prevention trials aimed at delaying or preventing symptom onset. If you take fluoxetine, paroxetine, or bupropion for depression, check trial eligibility carefully, as these are strong CYP2D6 inhibitors excluded from some trials. Your neurologist can switch you to sertraline or citalopram if needed. VMAT2 inhibitors for chorea are generally allowed at a stable dose. The Huntington's Disease Society of America provides resources and genetic counseling referrals.
Meanwhile, 47 Huntington Disease trials are recruiting
Whatever the FDA decides here, research on Huntington Disease does not stop. A few currently enrolling studies, US sites first:
Other drugs Trial Friend tracks for Huntington Disease
Get notified when new huntington-disease trials open or existing trials change status, add sites, or update eligibility.
Frequently asked questions
When will the FDA decide on AMT-130?
uniQure has disclosed a decision window of later this cycle for AMT-130 in Huntington's disease, without an exact date.
What is AMT-130 being reviewed for?
uniQure submitted a BLA for AMT-130 in Huntington's disease. One-time gene therapy delivered by neurosurgery into the striatum to silence the huntingtin gene; would be the first treatment shown to slow Huntington's disease. uniQure submitted the BLA for accelerated approval on September 2, 2026 and requested priority review; the FDA's filing decision is due within 60 days and no target action date has been disclosed. Holds Breakthrough Therapy, RMAT and Fast Track designations.
What happens after the AMT-130 decision?
If approved, availability is not immediate: specialty pharmacy setup, insurance review, and patient assistance typically take weeks even when everything goes right. If the FDA issues a complete response letter, the application was not approved in its current form; CRLs are often about manufacturing or data presentation rather than efficacy, and sponsors frequently resubmit. This page updates with the outcome either way.
Where this date comes from
The FDA does not publish PDUFA dates; companies disclose them. This one comes from uniQure SEC filing. Dates can move, and the FDA can act early or late. This page rechecks against our calendar, which is re-verified daily.